Restored motor function in mouse models of Parkinson’s disease  

Neuroscience Parkinson's disease

Gain Therapeutics has announced preclinical data demonstrating that its clinical-stage GCase regulator GT-02287 provided neuroprotection and restored motor function in Parkinson’s disease models following delayed administration.  

The data were accepted as a late-breaker abstract and was presented at the 20th Annual WORLDSymposium. 

“We believe the data showing complete restoration of motor function in a therapeutic model are remarkable and further support the potential of GT-02287 to slow or stop the progression of Parkinson’s disease, a disease for which only symptomatic treatments are available to patients at this time,” said Matthias Alder, Gain Therapeutics’ Chief Executive Officer.  “We are currently conducting a Phase I clinical trial of GT-02287 in healthy adults to evaluate its safety, tolerability, and pharmacokinetics, and plan to commence treatment of patients in an extension of that clinical trial in Q3 of this year.”  

The preclinical study showed that GT-02287 restored motor function in a mouse model, even following a delayed administration of the drug candidate after the initial toxic insult mimicking the effects of GBA1 Parkinson’s disease. Further, animals in the most challenging treatment group – those that began treatment eight days following onset of the disease – showed motor improvement from day 14-27, which suggests progressive reversal of neuronal deficit associated with continued treatment duration.   

Rescue of locomotor impairment was reflected in the complete reversal of plasma levels of Neurofilament Light Chain (NfL) to the level of the control arm in the study, which also suggests a neuroprotective effect of GT-02287. The emerging neurodegeneration biomarker NfL was previously accepted by the US Food and Drug Administration (FDA) for the accelerated approval of tofersen for the treatment of amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS) and was recommended by the FDA as an exploratory endpoint for neuronopathic mucopolysaccharidosis II (MPS II) clinical trials. 

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